A Study to Evaluate Safety and Exploratory Efficacy of KTP-001 in Subjects With Lumbar Disc Herniation

Overview

The purpose of this study is to evaluate the safety and exploratory efficacy of KTP-001 in subjects with lumbar disc herniation.

Full Title of Study: “Open-Label, Non-controlled, Single Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of KTP-001 in Subjects With Lumbar Disc Herniation”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: October 16, 2018

Detailed Description

This study was a first-in-human, open-label, non-controlled single ascending dose study of KTP-001 in male and female subjects between the ages of 30 and 70 years with a single herniated lumbar disc. After obtaining informed consent, subjects were evaluated during a screening period of no more than 3 weeks (21 days). This study was conducted in 10 centers in the US. Subjects that met all screening requirements and inclusion criteria and none of the exclusion criteria were enrolled into the study. Overall, 24 subjects were enrolled and treated: 6 subjects in each cohort. Cohort 1 received a 5 μg/disc dose of KTP-001 by intradiscal injection. Following administration of study drug, subjects were confined to the study center for 24 hours to collect data for safety and efficacy measures and collect blood samples for safety, PK evaluation, exploratory PD and anti-KTP-001 antibody and then returned for further assessments at various intervals from weeks 1 through to month 24. After all subjects in Cohort 1 had received study drug, safety measures were evaluated by a Data and Safety Monitoring Board (DSMB) to determine whether to escalate KTP-001 administration to the next dose level. If appropriate, Cohort 2 subjects received 15 μg/disc of KTP-001, Cohort 3 subjects received 50 μg/disc of KTP-001, and Cohort 4 subjects received 150 μg/disc of KTP-001 by intradiscal injection. All safety, PK, and exploratory efficacy assessments were performed for the subjects in the subsequent cohorts as were performed for Cohort 1.

Interventions

  • Drug: KTP-001
    • KTP-001 is one time dose intradiscally.

Arms, Groups and Cohorts

  • Experimental: Cohort 1
    • one time 5 μg/disc dose of KTP-001 by intradiscal injection
  • Experimental: Cohort 2
    • one time 15 μg/disc dose of KTP-001 by intradiscal injection
  • Experimental: Cohort 3
    • one time 50 μg/disc dose of KTP-001 by intradiscal injection
  • Experimental: Cohort 4
    • one time 150 μg/disc dose of KTP-001 by intradiscal injection

Clinical Trial Outcome Measures

Primary Measures

  • Safety Assessed by Adverse Events, Magnetic Resonance Imaging (MRI), X-ray Imaging, Physical Examination, Neurologic Examination and Vital Signs
    • Time Frame: 24 months
    • Any clinically significant changes were recorded as adverse events. They are described in the adverse events section of the results. AEs related to MRI, X-ray Imaging, Physical examination, and Neurologic examination are considered as adverse events of special interest (AESI). A treatment-emergent AE (TEAE) was defined as an AE that was not present prior to treatment with study drug, but appeared following treatment or was present at treatment initiation but worsened in severity during treatment.
  • Number of Participants With Change in 12-lead Electrocardiogram (ECG) and Clinical Laboratory Tests (CLT)
    • Time Frame: 13 weeks
    • Assessment of the number of participants with change in 12-lead ECG and CLT were assessed from baseline, 24 hours and 13 weeks.

Secondary Measures

  • Number of Participants Serum Concentrations of KTP-001 Below the Limit of Quantification (BLQ)
    • Time Frame: 13 weeks
    • The serum concentrations of KTP-001 were below the limit of quantification (BLQ) (<100 ng/mL) at all time points in all participants
  • Number of Participants With Anti-KTP-001 Antibody
    • Time Frame: 13 weeks

Participating in This Clinical Trial

Inclusion Criteria

  • Subject has had a single contained or noncontained (extruded) lumbar disc herniation (L3-L4, L4-L5 or L5-S1) diagnosed by clinical symptoms and/or physical findings and confirmed by MRI. – Subject has leg pain with a documented positive straight leg raise (SLR) test or femoral stretch test (FST). – Subject has experiences herniated disc symptoms for at least 6 weeks prior to the study without relief with pain medications and other therapies. – Subject has a BMI of 18 to 35 kg/m2 Exclusion Criteria:

  • Subject has a sequestered lumbar disc herniation or intrathecal herniation confirmed by MRI – Subject has two or more symptomatic lumbar disc herniations – Previous intradiscal therapeutic intervention or has had any lumbar surgery – Presence of lumbar spine disease and/or deformity other than a lumbar disc herniation – Active smoker or is unable to abstain from tobacco use for 2 weeks prior to study injection – Subject has a history or presence of significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, or psychiatric disease

Gender Eligibility: All

Minimum Age: 30 Years

Maximum Age: 70 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Teijin America, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.